Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program

ConditionsHereditary Transthyretin-mediated (hATTR) Amyloidosis Polyneuropathy

Trial statusRecruiting

Trial phaseNot listed

Trial typeObservational

Biological sexFemale

AgeNot listed

SponsorAlnylam Pharmaceuticals

About this trial

The purpose of this study is to collect and evaluate pregnancy outcomes, pregnancy complications, and fetal/neonatal/infant outcomes in women exposed to patisiran-LNP.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Documentation that the patient was exposed to patisiran-LNP at any point starting from 12 weeks before LMP or at any point during pregnancy

Germany Only: Patients must be treated per the summary of product characteristics (SmPC) for any approved treatment for ATTR amyloidosis

Disqualifiers

There are no exclusion criteria for participation in this program.

Trial population

Any woman exposed to patisiran-LNP 12 weeks prior to their last menstrual period (LMP) or at any time during pregnancy.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

Not listed

Trial groups

10 Participants

are grouped into 1 trial group

Group A: Patisiran

Trial outcomes

Primary outcomes

Prevalence of Major Congenital Malformations

Major congenital malformations will be classified according to the European Concerted Action on Congenital Anomalies and Twins (EUROCAT) and Metropolitan Atlanta Congenital Defects Program (MACDP) classification systems.

Time frame

From 12 weeks prior to last dose of patisiran through one year after birth (Up to 21 months)

Secondary outcomes

Prevalence of Minor Congenital Malformations

Minor congenital malformations will be classified classified according to the EUROCAT and MACDP classification systems.

Time frame

From 12 weeks prior to last dose of patisiran through one year after birth (Up to 21 months)

Prevalence of Pregnancy Outcomes

Pregnancy outcomes are defined as live birth, spontaneous abortions, stillbirths, elective abortions, molar or pregnancy, ectopic pregnancy, preterm births, and maternal death.

Time frame

From 12 weeks prior to last dose of patisiran through one year after birth (Up to 21 months)

Prevalence of Other Adverse Fetal/Neonatal/Infant Outcomes

Other adverse fetal/neonatal/infant outcomes are defined as low birth weight, failure to thrive, small for gestational age, postnatal growth and development, neonatal, and perinatal, or infant death.

Time frame

From 12 weeks prior to last dose of patisiran through one year after birth (Up to 21 months)